Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy

PURPOSE: SMT C1100 is a utrophin modulator being evaluated as a treatment for Duchenne muscular dystrophy (DMD). This study, the first in pediatric DMD patients, reports the safety, tolerability and PK parameters of single and multiple doses of SMT C1100, as well as analyze potential biomarkers of m...

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Veröffentlicht in:PLoS One
Hauptverfasser: Ricotti, Valeria, Spinty, Stefan, Roper, Helen, Hughes, Imelda, Tejura, Bina, Robinson, Neil, Layton, Gary, Davies, Kay, Muntoni, Francesco, Tinsley, Jonathon
Format: Artigo
Sprache:Inglês
Veröffentlicht: Public Library of Science 2016
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Research Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4824384/
https://ncbi.nlm.nih.gov/pubmed/27055247
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0152840
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